Track­ing a suc­cess­ful DL­B­CL as­sault in Phase II, Mor­phoSys sets out to seize quick OK for MOR208

En­cour­aged by a break­through des­ig­na­tion at the FDA and its lat­est batch of up­beat da­ta from a Phase II study of its an­ti-CD19 an­ti­body MOR208, Mor­phoSys says it’s blaz­ing a straight path to reg­u­la­tors in search of an ac­cel­er­at­ed ap­proval.

Mor­phoSys ex­ecs spent a good deal of time talk­ing to an­a­lysts yes­ter­day about their rea­sons for shoot­ing at a quick OK. The Ger­man biotech high­light­ed up­dat­ed da­ta demon­strat­ing that of the 68 evalu­able pa­tients in their study for dif­fuse large B cell lym­phoma — a pop­u­lar dis­ease tar­get in on­col­o­gy — they tracked a 49% over­all re­sponse rate and a 31% com­plete re­sponse rate.

They al­so have a pre­lim­i­nary pro­gres­sion free sur­vival rate of 50.5% at 12 months.

Malte Pe­ters

This is a sin­gle arm study of the Mor­phoSys an­ti­body com­bined with lenalido­mide for 81 DL­B­CL pa­tients not el­i­gi­ble for high-dose chemo or au­tol­o­gous stem cell trans­plan­ta­tion. And it says a lot about why biotechs are bar­rel­ing in­to can­cer with high ex­pec­ta­tions of break­ing new ground ear­li­er than ever.

Malte Pe­ters, the chief de­vel­op­ment of­fi­cer at Mor­phoSys, spelled it out for an­a­lysts:

Specif­i­cal­ly 29 out of 33 pa­tients which have re­spond­ed, that amount to 88% are on­go­ing, 20 of whom have an on­go­ing com­plete re­sponse. The mean time to re­spond was short with 1.8 months and the mean time to ex­pe­ri­ence a com­plete re­sponse was 3.6 months….

At this time, we are hop­ing to be able to sub­mit da­ta from the cur­rent L-MIND tri­al to the FDA as a ba­sis for reg­u­la­to­ry ap­proval. Our goal is to achieve an ac­cel­er­at­ed ap­proval based pri­mar­i­ly on the L-MIND study. Apart from L-MIND, we are cur­rent­ly in­ves­ti­gat­ing MOR208 in the same pa­tient pop­u­la­tion in our Phase III B-MIND tri­al. This is a head-to-head study in­ves­ti­gat­ing the ef­fi­ca­cy of MOR208 plus ben­damus­tine ver­sus rit­ux­imab plus ben­damus­tine. Rit­ux­imab plus ben­damus­tine is one of the most com­mon­ly used reg­i­mens in the re­lapse re­frac­to­ry DL­B­CL set­ting.

The FDA al­ready has this drug down in the spe­cial BTD path based on an ear­li­er snap­shot of da­ta from the same study. That gives Mor­phoSys a rea­son­able hope that it can move fast to­ward a fil­ing while lin­ing up the big­ger Phase III as a con­fir­ma­to­ry study.

Si­mon Mo­roney

At this point, the ac­cel­er­at­ed ap­proval path­way has been blazed by a mob of de­vel­op­ers who need­ed to show that they were do­ing a rea­son­able per­cent­age of late-stage pa­tients some good. The FDA has re­spond­ed with alacrity, and reg­u­la­tors in the US are on­ly pick­ing up even more speed un­der the di­rec­tion of FDA com­mis­sion­er Scott Got­tlieb. Mor­phoSys – al­lied with a group of A-list part­ners on oth­er drugs – has good rea­son to be­lieve that they can take the same short cut.

Not­ed CEO Si­mon Mo­roney:

“We are very en­cour­aged by our most re­cent clin­i­cal da­ta from the on­go­ing L-MIND tri­al, which sup­port our plan to de­vel­op MOR208 in com­bi­na­tion with lenalido­mide as a chemo-free treat­ment op­tion for this pa­tient pop­u­la­tion.”

Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,800+ biopharma pros reading Endpoints daily — and it's free.

We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.


ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology


ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development


CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,800+ biopharma pros reading Endpoints daily — and it's free.

Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,800+ biopharma pros reading Endpoints daily — and it's free.

SEC calls out lit­tle Ther­a­peu­tic­sMD for its in­sid­er con­tacts with an­a­lysts to boost share price, then halt rout

Back in May 2017, following an FDA rejection, TherapeuticsMD saw its share price plummet to the lowest levels in two years. The little Florida biotech eventually found its way back to the good side of regulators, scoring a curious OK a year later for its therapy preventing vaginal pain during sex. But the SEC is now accusing it of selectively disclosing nonpublic information in attempts to manipulate its stock.

In two instances in June and July of 2017, TherapeuticsMD allegedly violated the Regulation Fair Disclosure rule by sharing material information with certain sell-side analysts and not the public, resulting in a more favorable stock move than otherwise would be expected.

Endpoints News

Basic subscription required

Unlock this story instantly and join 57,800+ biopharma pros reading Endpoints daily — and it's free.

Therapists Marcela Ot'alora and Bruce Poulter are trained to conduct MDMA-assisted psychotherapy. In this reenactment, they demonstrate how they help guide and watch over a patient who is revisiting traumatic memories while under the influence of MDMA. (Photo: Multidisciplinary Association for Psychedelic Studies)

MD­MA, now in Phase III, shows promise as a PTSD treat­ment

The first time Lori Tipton tried MDMA, she was skeptical it would make a difference.

“I really was, at the beginning, very nervous,” Tipton said.

MDMA is the main ingredient in the club drug known as ecstasy or molly. But Tipton wasn’t taking pills sold on the street to get high. She was trying to treat her post-traumatic stress disorder by participating in a clinical trial.

After taking a dose of pure MDMA, Tipton lay in a quiet room with two specially trained psychotherapists. They sat next to her as she recalled some of her deepest traumas, such as discovering her mother’s body after Tipton’s mother killed two people and then herself in a murder-suicide.

Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin Bio Ventures and UBS Oncology Impact Fund in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.